A twice-yearly treatment for Neuromyelitis Optica Spectrum Disorder (NMOSD) has been found to reduce attacks and hospitalisations in adult patients, according to new trial data presented at the 8th Congress of the European Academy of Neurology (EAN) in Vienna.

Inebilizumab (Uplizna) received marketing authorization from the European Commission (EC) in April and is the first and only targeted CD19+ B-cell-depleting monotherapy proven to reduce NMOSD in patients who are anti-aquaporin-4 immunoglobulin G seropositive (AQP4-IgG+).

What is neuromyelitis optica spectrum disorder (NMOSD)?

NMOSD is a rare, severe, relapsing, neuroinflammatory autoimmune disease that attacks the optic nerve, spinal cord, brain and brain stem, often leading to vision loss and paralysis.

People with NMOSD live with unpredictable, repeat attacks, with 90% experiencing a secondary attack with the first five years of diagnosis.

The consequences of NMOSD extend beyond clinical impact and include physical, functional and psychological impacts on patients’ quality of life.

Women are nine times more likely to be impacted than men and the average age of onset is 40.

Families are therefore often significantly affected by NMOSD, with 60% of patients reporting that the disease affects their ability to work, perform physical activities and accomplish things all or most of the time.

The role of B cells in NMOSD

In NMOSD, damage is caused when CD19+ expressing B cell lymphocytes (plasmablasts and plasma cells) produce AQP4-IgG+, triggering an escalating autoimmune reaction.

Depletion of CD19+ B cells is thought to remove important contributors to autoimmune reactions, including inflammation, lesion formation or astrocyte damage.

Inebilizumab helps to reduce attacks in NMOSD patients by destroying autoimmune disease producing B cells whilst retaining protective B-cell immunity, which has been shown to have a distinct link to improved clinical outcomes in patients.

Study authors Friedemann Paul M.D and Max Delbrueck said: “With Uplizna, physicians have a treatment option that can be given twice a year after initial dosing to help prevent NMOSD attacks by specifically targeting CD19 B-cells, which play a central role in the pathogenesis of the disease.”

Inebilizumab halved the number of attacks compared to placebo

The trial data showed that patients treated with inebilizumab experienced fewer attacks (12.5%) compared to those treated with placebo (30%) as well as fewer NMOSD-related hospitalisations (mean, EU: 1.0 vs 2.0; non-EU: 1.0 vs 1.33).

Karl Boegl, M.D. Ph.D., executive director, EMEA regional medical affairs lead, Horizon, said: “The Uplizna pivotal trial is the largest in NMOSD and clearly demonstrates the merits of targeting CD19 B-cells, including plasmablasts and plasma cells, to provide broad, deep and durable B-cell depletion.”

He added that the data provides treating physicians with “greater certainty” that “a targeted monotherapy like Uplizna can be a valuable option for the treatment of NMOSD patients in Europe.”